The following is an excerpt from a press release from Genentech. Read the press release in its entirety here.
Genentech, a member of the Roche Group announced data from the primary analysis of the Phase III HAVEN 2 study evaluating Hemlibra® prophylaxis in children younger than 12 years of age with hemophilia A with factor VIII inhibitors, including longer follow-up for once-weekly dosing and new data for less frequent dosing schedules (every two weeks or every four weeks). These data from the largest pivotal study in children with hemophilia A with factor VIII inhibitors were presented at the 60th American Society of Hematology Annual Meeting.
“Children with inhibitors are at increased risk of life-threatening bleeds and may experience frequent, repeated bleeding into joints,” said Guy Young, M.D., director of Hemostasis and Thrombosis Center, Children’s Hospital Los Angeles, and professor of Pediatrics, University of Southern California Keck School of Medicine, Los Angeles, California. “These updated data from HAVEN 2 showed that the majority of children with hemophilia A with factor VIII inhibitors treated with emicizumab-kxwh had zero treated bleeds across three different dosing schedules, reinforcing the ability of this medicine to provide sustained, effective bleed control.”
In updated results from the HAVEN 2 study with a median of 11 additional months of data, 76.9 percent (95 percent CI: 64.8; 86.5) of children with hemophilia A with factor VIII inhibitors treated with Hemlibra once weekly (n=65) experienced zero treated bleeds. Importantly, once-weekly Hemlibra showed a 99 percent (95 percent CI: 97.7; 99.4) reduction in treated bleeds compared to prior treatment with bypassing agents (BPAs) as prophylaxis (n=15) or on-demand (n=3) in a prospective intra-patient comparison. New data also showed that 90 percent (95 percent CI: 55.5; 99.7) of children with factor VIII inhibitors receiving Hemlibra every two weeks (n=10) and 60 percent (95 percent CI: 26.2; 87.8) of children receiving Hemlibra every four weeks (n=10) experienced zero treated bleeds, demonstrating clinically meaningful bleed control at both dosing schedules. No cases of thrombotic microangiopathy (TMA) or thrombotic events occurred. The most common adverse events (AEs) in the HAVEN 2 study primary analysis were consistent with those previously observed in the interim analyses.
“The updated analysis from the HAVEN 2 study supports the potential of Hemlibra to control bleeds at less frequent subcutaneous dosing, providing parents and their children more flexibility to choose a treatment schedule that is right for them,” said Sandra Horning, M.D., chief medical officer and head of Global Product Development. “Many children with hemophilia A with factor VIII inhibitors have already experienced the benefits of Hemlibra, and with these new positive data, we are confident that this treatment will continue to make a meaningful difference in their lives.”
Read the press release in its entirety here.